FDA de­rails Acor­da's mar­ket­ing pitch, cre­at­ing a cri­sis as flag­ship drug founders

Acor­da just en­tered a world of hurt.

With its patent pro­tec­tion crum­bling around its main­stay drug, the FDA has re­fused to file its ap­pli­ca­tion for CVT-301, the res­cue pro­gram that was des­ig­nat­ed to take the lead drug’s place. The RTF cre­ates an em­bar­rass­ing and per­haps crip­pling prob­lem for the biotech, which is fight­ing a last-ditch bat­tle to main­tain patent pro­tec­tion on its one big drug, Ampyra.

Ron Co­hen

Ac­cord­ing to Acor­da, the FDA says the com­pa­ny’s mar­ket­ing ap­pli­ca­tion, rushed to reg­u­la­tors as the com­pa­ny re­struc­tured and down­sized, is in­com­plete. Reg­u­la­tors said they were un­able to do a prop­er re­view as they don’t know what date Acor­da’s man­u­fac­tur­ing cen­ter will be ready for in­spec­tion and have a “ques­tion re­gard­ing the sub­mis­sion of the drug mas­ter pro­duc­tion record.”

Acor­da re­port­ed that the FDA al­so re­quest­ed ad­di­tion­al in­for­ma­tion at re­sub­mis­sion, which was not part of the ba­sis for the RTF.

Its shares quick­ly plunged 26% on the news.

Nor­mal­ly, a de­lay like this would be seen as a sticky prob­lem, but one that could po­ten­tial­ly be re­solved with­out huge de­lays. The prob­lem for Acor­da, though, is that the com­pa­ny has vir­tu­al­ly no lee­way for a sna­fu like this. Not on­ly does it leave Acor­da’s ex­ec­u­tive team with a black eye on ex­e­cu­tion, it cre­ates se­ri­ous doubts about its abil­i­ty to quick­ly and ef­fi­cient­ly line up a re­place­ment drug for its sales team by next sum­mer, as the lit­i­ga­tion over its patents for Ampyra comes to a head.

Leerink’s Paul Mat­teis found the whole thing puz­zling and dis­con­cert­ing, con­sid­er­ing the like­li­hood of a loom­ing ap­proval. And he still has ques­tions about what is go­ing on at Acor­da.

It seems like the best case sce­nario would be a re-sub­mis­sion around the end of this year, and its en­cour­ag­ing that FDA did not make any re­quests re­gard­ing the ef­fi­ca­cy or safe­ty da­ta. Nonethe­less, even af­ter speak­ing with the com­pa­ny, the RTF is mys­te­ri­ous to us, and we ex­pect the stock to be un­der pres­sure un­til opac­i­ty is lift­ed.

As I re­port­ed in June, while re­view­ing the late-stage da­ta sub­mit­ted to the FDA on the in­haled lev­odopa, any set­back on CVT-301 would be a cat­a­stro­phe for Acor­da.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

Now Acor­da is left to des­per­ate­ly seek a quick meet­ing with reg­u­la­tors to clear up any of these ques­tions. But the FDA is not known for scram­bling a re­sponse in any of these sit­u­a­tions, leav­ing Acor­da’s time­line in the air just when it most needs cer­tain­ty. Just weeks ago one of its biggest in­vestors pushed CEO Ron Co­hen to auc­tion the com­pa­ny off rather than risk the con­se­quences of fail­ing at its high wire act. The com­pa­ny pushed back, but a sale may come back on the ta­ble now whether or not it likes the idea.

“We will work with the FDA as quick­ly as pos­si­ble to ad­dress the open is­sues and to clar­i­fy the path to suc­cess­ful­ly re-file our ap­pli­ca­tion,” said Co­hen in a state­ment. “We re­main con­fi­dent in IN­BRI­JA’s da­ta pack­age and its promise as an im­por­tant new ther­a­py for peo­ple with Parkin­son’s dis­ease. We see tremen­dous long-term val­ue in its sol­id clin­i­cal pro­file, sig­nif­i­cant com­mer­cial op­por­tu­ni­ty and strong IP, and we re­main fo­cused on work­ing to bring pa­tients this im­por­tant new ther­a­py.”

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Ac­celeron of­fers thumbs up on a PhII suc­cess for would-be block­buster drug -- but holds da­ta in re­serve

There’s no public data yet, but Acceleron $XLRN says that its first major trial readout of 2020 is a success.

In a Phase II study of 106 patients with pulmonary arterial hypertension (PAH), Acceleron’s experimental drug sotatercept hit its primary endpoint: a significant reduction in pulmonary vascular resistance. The drug also met three different secondary endpoints, including the 6-minute walking test.

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Patrik Jonsson, the president of Lilly Bio-Medicines

Who knew? Der­mi­ra’s board kept watch as its stock price tracked Eli Lil­ly’s se­cret bid­ding on a $1.1B buy­out

In just 8 days, from December 6 to December 14, the stock jumped from $7.88 to $12.70 — just under the initial $13 bid. There was no hard news about the company that would explain a rise like that tracking closely to the bid offer, raising the obvious question of whether insider info has leaked out to traders.

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Short at­tack­er Sahm Ad­ran­gi draws crosshairs over a fa­vorite of Sanofi’s new CEO — with PhII da­ta loom­ing

Sahm Adrang Kerrisdale

Kerrisdale chief Sahm Adrangi took a lengthy break from his series of biotech short attacks after his chief analyst in the field pulled up stakes and went solo. But he’s making a return to drug development this morning, drawing crosshairs over a company that’s one of new Sanofi CEO Paul Hudson’s favorite collaborators.

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Civi­ca and Blue Cross Blue Shield launch new ven­ture to low­er gener­ic prices

Five years after Martin Shkreli put a smug face to the volatile prices companies can charge even for generic drugs, payers and governments are coming up with outside-the-box solutions.

The latest fix is a new venture from the Blue Cross Blue Shield Association, 18 of its members and Civica, the generics company founded in 2018 by hospitals fed up with high prices for drugs that had long-since lost patent protection. While Civica focused on drugs that hospitals purchased, the new company will aim to lower prices on drugs that, like Shkreli’s Daraprim, are purchased by individuals.

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Merck Invests in State-Of-The-Art Biotech Development Facility in Switzerland

Mer­ck KGaA match­es lofty R&D goals with €250M in­vest­ment in­to a new clin­i­cal man­u­fac­tur­ing site in Switzer­land

As Merck KGaA strives to prove itself as a capable biopharma R&D player, it has begun construction on a €250 million facility dedicated to developing and manufacturing drugs for use in clinical trials.

The German drugmaker chose a location at Corsier-sur-Vevey, Switzerland, where it already has a commercial manufacturing site, in order to “bridge together research and manufacturing.”

“This investment in the Merck Biotech Development Center reflects our commitment to speed up the availability of new medicines for patients in need, and confirms the importance of Switzerland as our prime hub for the manufacturing of biotech medicines,” CEO Stefan Oschmann said at the groundbreaking ceremony, according to a statement.

Breast can­cer ap­proval in tow, As­traZeneca, Dai­ichi armed an­ti­body scores in key gas­tric can­cer study

AstraZeneca kicked off Monday with a flurry of good news. Apart from unveiling positive results on its stroke trial testing its clot-fighter Brilinta, and welcoming its experimental IL-23 inhibitor brazikumab back from Allergan — the British drugmaker also disclosed some upbeat gastric cancer data on its HER2-positive oncology therapy it is collaborating on with Daiichi Sankyo.

Buoyed by the performance of its oncology drugs, last March AstraZeneca chief Pascal Soriot bet big to partner with Daiichi on the cancer drug, with $1.35 billion upfront in a deal worth up to roughly $7 billion. Roughly 8 months later, as 2019 drew to a close, the FDA swiftly approved the drug — trastuzumab deruxtecan — for use in breast cancer, months ahead of the expected decision date.

Sor­ren­to shrugs off an anony­mous pri­vate eq­ui­ty group’s $1B of­fer to buy the com­pa­ny

San Diego-based Sorrento Therapeutics isn’t going the M&A route — at least not today.

The biotech caused quite a stir when it put out word a few weeks ago that an unidentified private equity group was bidding a billion dollars-plus for the company. The news drove a quick spike in the company’s share price as investors hooked up for the ride — that didn’t happen.

The update sparked a 5% drop in the share price $SRNE ahead of the bell. It’s now trading just above $4, without any evidence that the $7 price looked like it was firm.

UP­DAT­ED: Ab­b­Vie do­nates $1M+ of the HIV drug that Chi­na is now rec­om­mend­ing for coro­n­avirus treat­ment

AbbVie is donating more than $1 million worth of an HIV drug to help combat the fast-spreading coronavirus outbreak in China, the company announced on Friday.

China’s National Health Commission has suggested Aluvia, a pill containing lopinavir and ritonavir, as one of two possible treatments for the symptoms of the virus currently known as 2019-nCoV in the absence of effective antiviral medications. The other part is nebulized alpha-interferon.